Copenhagen, 4th July 2007
Santaris Pharma announced today that the Danish National Advanced Technology Foundation (ATF) has awarded a grant of €1.35 million towards the development of the first ever drug targeted against a human microRNA. The drug candidate, SPC3649, is an antagonist of the liver-specific microRNA-122 and is being developed by Santaris in collaboration with the Centre for Biological Sequence Analyses at the Technical University of Denmark and the Department of Cellular and Molecular Medicine, University of Copenhagen. This is the second award from the Danish ATF to the Santaris Pharma led consortium. In 2006 the ATF made a grant to Santaris and the University of Copenhagen of €1.3m, to support research in the rapidly evolving field of microRNA therapeutics.
MicroRNAs are a newly discovered class of tiny regulatory molecules which appear to control many biological processes within cells. There is growing evidence that microRNAs are also associated with the onset and progression of many diseases. Santaris Pharma’s new drug candidate, SPC3649, targets a particular liver-expressed microRNA called miR-122. Treatment of monkeys with the drug for only a few days results in a long-lasting reduction of blood cholesterol. In addition, the drug blocks Hepatitis C virus growth in human liver cells. Santaris Pharma is developing the compound as a potential new medicine for hyperlipidaemia and Hepatitis C infection.
Announcing the award, Sakari Kauppinen, Santaris Pharma’s Associate Director of MicroRNA Research, commented:
"The entire field of molecular biology and medicine has been thrown wide open in recent years by the discovery of microRNAs. Santaris Pharma’s LNA technology has unique advantages in developing potential breakthrough drugs acting on disease-associated microRNAs. We are delighted that the Danish Advanced Technology Foundation has recognised the scientific and commercial potential of our world leading position in this field. SPC3649 may well become the first microRNA antagonist drug to be tested in human clinical trials."
For further information, please contact:
Randi Krogsgaard
Director of Communications, Santaris Pharma
Tel +45 45179879 (direct) +45 4517 9820 (Company)
Mobile/Cell +45 20488384 E-mail: rmk@santaris.com
Dr. Sakari Kauppinen
Associate Director, MicroRNA Research, Santaris Pharma
Tel. +45 45 17 98 38 (direct) +45 4517 9820 (Company)
Mobile/Cell +45 20 47 79 94 E-mail: sk@santaris.com
Forward Looking Statements
This written announcement contains forward-looking statements, identified by the use of forward-looking words such as "believes," "expects," "may," "will," "should", "potential," "anticipates," "plans" or "intends" and similar expressions. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual results, events or developments to be materially different from the future results, events or developments indicated in such forward-looking statements. Such factors include, but are not limited to the timing, success and cost of clinical studies; the ability to obtain regulatory approval of products, market acceptance of and future demand for Santaris products and the impact of competitive products and pricing. These factors should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. No assurance can be given that the future results covered by the forward-looking statements will be achieved. All information in this press release is as of the date of this press release and Santaris does not intend to update this information.
NOTES FOR EDITORS
About SPC3649
SPC3649 is a specific antagonist of microRNA-122 (miR-122), which is expressed in the liver, where it is involved in establishing and maintaining a variety of liver cell-specific biological functions, including cholesterol, fatty acid and lipid metabolism. It has also been shown that miR-122 interacts with the hepatitis C virus genome facilitating viral replication in the host cell. The new Santaris drug candidate, SPC3649, binds specifically to microRNA-122 and prevents it from exerting its biological function. Blocking miR-122 function in this way appears to cause no obvious abnormality in normal liver tissue. However, treatment results in reduced plasma cholesterol levels. Systemic administration by intravenous injection of SPC3649 decreases plasma cholesterol levels by approximately 30-40% compared to saline-treated control animals. When administered to high fat-fed animals, SPC3649 normalizes blood cholesterol levels without the side effects of fatty liver seen with some other agents.
About Santaris Pharma
Santaris Pharma is a clinical-stage biopharmaceutical company focused on developing a new class of drugs intended to switch off the expression of harmful genes. Called RNA Antagonists, these new drugs are being developed by Santaris and its corporate partners for the treatment of cancer and metabolic disorders. Created in May 2003 and backed by a broad group of leading international life science venture capital investors, Santaris Pharma completed a Euro 40m second round of equity financing in May 2006. In July 2006, the Company entered into a global partnership with Enzon Pharmaceuticals of New Jersey to co-develop and commercialise a series of Santaris RNA Antagonists for improving the treatment of cancer.
Santaris Pharma’s RNA Antagonist drug pipeline is based on its unique LNA technology. LNA drugs, with their high potency and biostability, have the potential to transform the field of RNAi medicines, making specific and effective gene silencing a reality in human medicine. If this potential is realised, even in part, it may be possible to design new drugs to treat the underlying genetic causes of disease rather than just the physical symptoms. Santaris Pharma holds the world wide patent rights to the exploitation of LNA in pharmaceuticals and presently has three drugs in preclinical or clinical development. The lead drug candidate, SPC2996, is currently undergoing international, multicenter, phase I/II clinical studies in Chronic Lymphocytic Leukemia (CLL).
For further company information see
www.santaris.com